创建多中心罕见病联盟--嗜酸性胃肠病研究人员联盟(CEGIR)。

Katherine Cheng, Sandeep K Gupta, Susanna Kantor, Jonathan T Kuhl, Seema S Aceves, Peter A Bonis, Kelley E Capocelli, Christina Carpenter, Mirna Chehade, Margaret H Collins, Evan S Dellon, Gary W Falk, Rashmi Gopal-Srivastava, Nirmala Gonsalves, Ikuo Hirano, Eileen C King, John Leung, Jeffrey P Krischer, Vincent A Mukkada, Alain Schoepfer, Jonathan M Spergel, Alex Straumann, Guang-Yu Yang, Glenn T Furuta, Marc E Rothenberg
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摘要

嗜酸性粒细胞胃肠病(EGIDs)影响胃肠道的各个部分。由于这些疾病非常罕见,因此必须开展合作,将受试者纳入临床研究并对更广泛的人群进行研究。罕见病临床研究网络(RDCRN)是美国国家转化科学促进中心(NCATS)的一项计划,该计划于 2014 年资助了嗜酸性胃肠病研究人员联合会(CEGIR),以推动 EGIDs 领域的发展。CEGIR促进了各中心、亚专科、患者、专业组织和患者权益团体之间的合作,包括14个临床研究基地。它已成功启动了两项大型多中心临床研究,旨在完善 EGID 诊断和管理。目前正在开展几项试点研究,重点关注 EGID 的各个方面,包括新型治疗干预措施、诊断和监测方法以及微生物组在发病机制中的作用。目前,CEGIR 通过职业培训发展计划培养了五名医生学者,自成立以来已发表 40 多篇手稿。本综述重点介绍 CEGIR 的运作模式和进展,以及它如何促进思想交流框架并激励研究和创新。该联盟为其他潜在临床领域的进展提供了一个模式。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Creating a multi-center rare disease consortium - the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR).

 Eosinophilic gastrointestinal disorders (EGIDs) affect various segments of the gastrointestinal tract. Since these disorders are rare, collaboration is essential to enroll subjects in clinical studies and study the broader population. The Rare Diseases Clinical Research Network (RDCRN), a program of the National Center for Advancing Translational Sciences (NCATS), funded the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) in 2014 to advance the field of EGIDs. CEGIR facilitates collaboration among various centers, subspecialties, patients, professional organizations and patient-advocacy groups and includes 14 clinical sites. It has successfully initiated two large multi-center clinical studies looking to refine EGID diagnoses and management. Several pilot studies are underway that focus on various aspects of EGIDs including novel therapeutic interventions, diagnostic and monitoring methods, and the role of the microbiome in pathogenesis. CEGIR currently nurtures five physician-scholars through a career training development program and has published more than 40 manuscripts since its inception. This review focuses on CEGIR's operating model and progress and how it facilitates a framework for exchange of ideas and stimulates research and innovation. This consortium provides a model for progress on other potential clinical areas.

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